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MEK Inhibitor
AZD6244 for Neurofibromatosis
Phase 1 & 2
Waitlist Available
Led By Brigitte C Widemann, M.D.
Research Sponsored by National Cancer Institute (NCI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Performance status: Karnofsky performance level >70% for patients >16 years old, Lansky performance >70% for children <16 years old
Renal function: creatinine clearance or GFR >=60ml/min/1.73 m(2) or normal serum creatinine based on age
Must not have
Inability to swallow capsules
Ongoing radiation therapy, chemotherapy, hormonal therapy, immunotherapy, or biologic therapy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up before cycles 3,5,9,13 and then every 12 cycles
Awards & highlights
No Placebo-Only Group
Summary
This trial is testing a drug to see if it can prevent, slow down the growth, or shrink tumors in children and young adults with plexiform neurofibromas.
Who is the study for?
This trial is for children and young adults aged 2 to 18 with inoperable plexiform neurofibromas, a type of nerve tumor. Participants must be able to swallow capsules, have no severe allergies to the drug's ingredients, and not require surgery within the first three months. They should also have normal organ function and not be on certain other medications or treatments.
What is being tested?
The study tests AZD6244 hydrogen sulfate's ability to stop growth or shrink inoperable plexiform neurofibromas in children. The treatment involves taking the drug twice daily over cycles of 28 days with regular health checks, blood tests, urine tests, and imaging studies.
What are the potential side effects?
Potential side effects may include skin sensitivity requiring sun avoidance and sunscreen use; digestive issues that could affect absorption; increased risk if there are existing conditions like uncontrolled diseases or infections; possible impact on vision; and potential interaction with grapefruit products.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am mostly active and can care for myself.
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My kidney function is normal or only mildly impaired.
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I have been diagnosed with or tested positive for NF1.
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My blood pressure is within the normal range for my age, height, and gender.
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My liver tests are within the required range.
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My heart pumps well and my heart rhythm is normal.
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I am between 3 and 18 years old and can swallow capsules.
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I am between 2 and 18 years old, can swallow capsules, and my body surface area is at least 0.55 m2.
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I have NF1 with tumors that can't be removed and may cause serious health problems.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I cannot swallow pills.
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I am currently receiving cancer treatment such as radiation, chemotherapy, or immunotherapy.
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I do not have severe illness, active infections, bleeding disorders, kidney transplant, hepatitis C, or HIV.
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I have ongoing nausea, vomiting, or serious gut issues.
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I have previously been treated with a MEK1/2 inhibitor.
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I have been diagnosed with a specific type of cancer.
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My blood pressure is not well controlled.
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I have ongoing side effects from anti-NF1 treatment.
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I need surgery within the first three treatment cycles.
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I have a known heart condition.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ first course
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~first course
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Ph1: Determine MTD and extended tolerability
Ph2: Evaluate the confirmed partial and complete response rate of selumetinib in children and young adults with NF1 and inoperable PN.
Secondary study objectives
Define toxicities
Measure adherence of chronic dosing
Ph2: Evaluate confirmed partial and complete response rate, and duration of response.
+4 moreSide effects data
From 2020 Phase 2 trial • 334 Patients • NCT0124824750%
Rash Acneiform
36%
Diarrhea
32%
Nausea
32%
Dry skin
27%
Anorexia
23%
Increased blood bilirubin
23%
Vomiting
23%
Increased AST
14%
Oral mucositis
14%
Weight loss
14%
Other skin and subcutaneous tissue disorders, specify
14%
Rash Maculopapular
14%
Epistaxis
9%
Fatigue
9%
Hyperglycemia
9%
Increased alkaline phosphatase
5%
Lung infection
5%
Dry mouth
5%
Pruritus
5%
Hyponatremia
5%
Dyspnea
5%
Palmar-plantar erythrodysesthesia syndrome
5%
Multi-organ failure
5%
Hypoalbuminemia
5%
Dysgeusia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm 1 - Erlotinib
Arm 3 - AZD6244 + MK-2206
Arm 4 - Sorafenib
Arm 2 - Erlotinib + MK-2206
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
2Treatment groups
Experimental Treatment
Group I: Arm 2Experimental Treatment1 Intervention
Phase 2: AZD6244 PO BID x 28 DAYS
Group II: Arm 1Experimental Treatment1 Intervention
Phase 1: AZD6244 PO BID x 28 DAYS
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AZD6244
2011
Completed Phase 2
~2220
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)Lead Sponsor
13,958 Previous Clinical Trials
41,112,481 Total Patients Enrolled
3 Trials studying Neurofibromatosis
290 Patients Enrolled for Neurofibromatosis
Brigitte C Widemann, M.D.Principal InvestigatorNational Cancer Institute (NCI)
20 Previous Clinical Trials
3,511 Total Patients Enrolled
1 Trials studying Neurofibromatosis
10 Patients Enrolled for Neurofibromatosis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I cannot swallow pills.I am currently receiving cancer treatment such as radiation, chemotherapy, or immunotherapy.I am mostly active and can care for myself.My kidney function is normal or only mildly impaired.I do not have severe illness, active infections, bleeding disorders, kidney transplant, hepatitis C, or HIV.I have been diagnosed with or tested positive for NF1.My blood pressure is within the normal range for my age, height, and gender.My liver tests are within the required range.My heart pumps well and my heart rhythm is normal.I have ongoing nausea, vomiting, or serious gut issues.I have previously been treated with a MEK1/2 inhibitor.I have been diagnosed with a specific type of cancer.I am not taking any medications that could affect the study drugs.I am between 3 and 18 years old and can swallow capsules.I am between 2 and 18 years old, can swallow capsules, and my body surface area is at least 0.55 m2.I have NF1 with tumors that can't be removed and may cause serious health problems.My blood pressure is not well controlled.I have an eye condition.I have not had major surgery in the last 4 weeks.I have ongoing side effects from anti-NF1 treatment.I need surgery within the first three treatment cycles.I have a known heart condition.
Research Study Groups:
This trial has the following groups:- Group 1: Arm 1
- Group 2: Arm 2
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.